Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project ...
Nov 18, 2020
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New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy. These findings, which emphasize the need for mobilization resistant AAV vectors, are ...
Nov 13, 2020
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Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver. This dietary induction of hepatocyte regeneration may be a viable clinical strategy ...
Nov 10, 2020
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The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible ...
Oct 9, 2020
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Gene therapy was successfully used to overcome the cardiac effects of Freidreich's ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy.
Sep 18, 2020
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A rational design approach created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties in the mouse retina and cornea. as reported in the peer-reviewed journal Human Gene Therapy.
Aug 14, 2020
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Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival ...
Aug 11, 2020
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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
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An initial trial in patients indicates that a new genetic treatment for complete color blindness, developed by research groups based in Tübingen and Munich is safe. Preliminary evidence for its efficacy has also been obtained.
May 1, 2020
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In an unprecedented effort, hundreds of thousands of researchers and clinicians worldwide are locked in a race against time to develop cures, vaccines, and better diagnostic tests for COVID-19, the illness caused by the virus ...
Apr 24, 2020
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